The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: 'The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis'
| Autor: | Miguel Sampayo-Cordero, Bernat Miguel-Huguet, Andrea Malfettone, José Manuel Pérez-García, Antonio Llombart-Cussac, Javier Cortés, Almudena Pardo, Jordi Pérez-López |
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| Přispěvatelé: | Institut Català de la Salut, [Sampayo-Cordero M, Malfettone A] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. [Miguel-Huguet B] Colorectal Unit, Department of Surgery, Hospital de Bellvitge, Barcelona, Spain. [Pérez-García JM] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. IOB Institute of Oncology, Quiron Salud Group, Madrid, Spain. [Llombart-Cussac A] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. Hospital Arnau de Vilanova, Universidad Católica de Valencia San Vicente Mártir, Valencia, Spain. [Cortés J] Medica Scientia Innovation Research (MedSIR), Barcelona, Spain. IOB Institute of Oncology, Quiron Salud Group, Madrid, Spain. Vall d’Hebron Institute of Oncology (VHIO), Barcelona, Spain. [Pérez-López J] Servei de Medicina Interna, Vall d’Hebron Hospital Universitari, Barcelona, Spain, Vall d'Hebron Barcelona Hospital Campus |
| Rok vydání: | 2021 |
| Předmět: |
0301 basic medicine
Other subheadings::/methods [Other subheadings] Review Disease Biochemistry 0302 clinical medicine afecciones patológicas signos y síntomas::procesos patológicos::atributos de la enfermedad::enfermedades raras [ENFERMEDADES] Clinical trials systematic review Otros calificadores::/métodos [Otros calificadores] case reports nonrandomized study Clinical endpoint Molecular Biosciences Biology (General) terapéutica::farmacoterapia::terapia enzimática::tratamiento de sustitución enzimática [TÉCNICAS Y EQUIPOS ANALÍTICOS DIAGNÓSTICOS Y TERAPÉUTICOS] media_common mucopolysaccharidosis Enzyme replacement therapy Rare diseases Systematic review Mucopolisacàrids Meta-analysis Malalties rares enzyme replacement therapy medicine.medical_specialty QH301-705.5 media_common.quotation_subject rare disease Mucopolysaccharides Biochemistry Genetics and Molecular Biology (miscellaneous) 03 medical and health sciences medicine Generalizability theory Intensive care medicine Molecular Biology Selection bias business.industry Pathological Conditions Signs and Symptoms::Pathologic Processes::Disease Attributes::Rare Diseases [DISEASES] Enzims - Ús terapèutic diagnóstico::pronóstico::resultado del tratamiento [TÉCNICAS Y EQUIPOS ANALÍTICOS DIAGNÓSTICOS Y TERAPÉUTICOS] Diagnosis::Prognosis::Treatment Outcome [ANALYTICAL DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT] meta-analysis 030104 developmental biology Avaluació de resultats (Assistència sanitària) business 030217 neurology & neurosurgery Therapeutics::Drug Therapy::Enzyme Therapy::Enzyme Replacement Therapy [ANALYTICAL DIAGNOSTIC AND THERAPEUTIC TECHNIQUES AND EQUIPMENT] Rare disease Assaigs clínics |
| Zdroj: | Dipòsit Digital de la UB Universidad de Barcelona Scientia Frontiers in Molecular Biosciences, Vol 8 (2021) Frontiers in Molecular Biosciences |
| Popis: | Estudi no aleatoritzat; Malaltia rara; Revisió sistemàtica Estudio no aleatorizado; Enfermedad rara; Revisión sistemática Nonrandomized study; Rare disease; Systematic review Nonrandomized studies are usually excluded from systematic reviews. This could lead to loss of a considerable amount of information on rare diseases. In this article, we explore the impact of excluding nonrandomized studies on the generalizability of meta-analyses results on mucopolysaccharidosis (MPS) disease. A comprehensive search of systematic reviews on MPS patients up to May 2020 was carried out (CRD42020191217). The primary endpoint was the rate of patients excluded from systematic reviews if only randomized studies were considered. Secondary outcomes included the differences in patient and study characteristics between randomized and nonrandomized studies, the methods used to combine data from studies with different designs, and the number of patients excluded from systematic reviews if case reports were not considered. More than 50% of the patients analyzed have been recruited in nonrandomized studies. Patient characteristics, duration of follow-up, and the clinical outcomes evaluated differ between the randomized and nonrandomized studies. There are feasible strategies to combine the data from different randomized and nonrandomized designs. The analyses suggest the relevance of including case reports in the systematic reviews, since the smaller the number of patients in the reference population, the larger the selection bias associated to excluding case reports. Our results recommend including nonrandomized studies in the systematic reviews of MPS to increase the representativeness of the results and to avoid a selection bias. The recommendations obtained from this study should be considered when conducting systematic reviews on rare diseases. |
| Databáze: | OpenAIRE |
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